We emphasize that this procedure, called retrovirus mediated gene transfer, has only been used before 
in about 50 patients. Because this procedure is new, it is possible that despite our extensive efforts 
other unforeseen problems may occur including the very remote possibility that death may occur. 
BENEFITS 
At the current point in time, we do not know whether you will have any benefit from participating in 
this study. If we are successful in inserting the GC gene in a sufficient number of your blood stem cells 
such that part of your blood cells start producing GC enzyme, this could lead to improvement of your 
disease. If you are receiving enzyme treatment and we can find cells in your blood which produce GC 
enzyme, our plan would be to slowly withdraw enzyme treatment over a period of 9 months to see 
jj whether withdrawal of enzyme replacement can be tolerated. Currently there is no certainty that the 
gene therapy approach being used in this study will be successful. It is possible, however, that results of 
I this study will lead to a new therapy which may benefit you in the future. 
:i 
ALTERNATIVES 
The alternatives are not to participate in this research study. 
OTHER INFORMATION 
Your decision to participate in this study is voluntary. You can decide whether or not to participate in 
this study. You may decide not to participate in this study at any time, for any reason, without notice. 
You will continue to receive medical care even if you decide to discontinue treatment on this study, 
r You will not be charged nor will you be paid for your participation in this study, although you will be 
charged for the medical and hospital care you require independent of this study just as though you were 
not part of this study. 
In order to evaluate the results of this study, your medical records will need to be available, now and in 
the future, to other physicians and researchers associated with the research project. The National 
Institutes of Health, the Food and Drug Administration will also have access to this information. All 
precautions to maintain confidentiality of medical records and personal information will be taken. Your 
personal identity will not be revealed in any publication or results. Study records will be maintained 
indefinitely for the purpose of analysis and follow-up. 
If you have questions about the research or a research related injury, please contact Friedrich 
Schuening, M.D. (667-4410) or your attending physician. 
If you have any questions about your rights as a research participant, please contact Karen Hansen in 
the Institutional Review Office of Fred Hutchinson Cancer Research Center at 206/667-4867. 
Investigator’s Statement 
I have provided an explanation of the above research program. The subject was given an opportunity to 
discuss the procedures, including possible alternatives, and to ask any additional questions. A signed 
I copy of this consent form has been given to the subject. 
I 
] ; ; 
Investigator’s Signature /Date 
Recombinant DNA Research, Volume 18 
[685] 
